Adding apatorsen to gemcitabine and nab-paclitaxel does not improve clinical outcomes among unselected patients with pancreatic cancer, according to research published in The Oncologist.1

Apatorsen is an oligonucleotide that targets heat shock protein 27 (Hsp27), which is associated with chemotherapy resistance. For this phase 2 randomized study ( Identifier: NCT01844817), researchers evaluated whether adding apatorsen to a standard chemotherapy regimen would improve outcomes among patients with metastatic pancreatic cancer, which carries a dismal prognosis of less than a year.

Of 132 enrolled patients, 66 were randomly assigned to receive gemcitabine, nab-paclitaxel, and apatorsen, or receive to gemcitabine, nab-paclitaxel, and placebo. Patients were treated until progression or intolerable toxicity.

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Overall responses were similar, with an objective response rate of 18% in both groups. Overall survival (OS) and progression-free survival (PFS) rates were 2.7 and 5.3 months, respectively, in the experimental arm, vs 3.8 and 6.9 months, respectively, for the control arm. Toxicity was similar in both arms.

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Among patients with high serum levels of Hsp27, the authors noted that apatorsen improved PFS (3.3 months) and OS (3.3 months) vs placebo (.9 months and 1 month, respectively).

The authors concluded that further studies of apatorsen “in unselected patients do not appear to be indicated, although the findings from this trial do hint at the possible prognostic and predictive value of serum Hsp27 that may warrant further investigation.”


  1. Ko AH, Murphy PB, Peyton JD, et al. A randomized, double‐blinded, phase II trial of gemcitabine and nab‐paclitaxel plus apatorsen or placebo in patients with metastatic pancreatic cancer: the RAINIER trial. Oncologist. 2017 Sep 21. doi: 10.1634/theoncologist.2017-0066 [Epub ahead of print]